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2016 – A year in review for Advanced Therapies

Guest post by Patricia Reilly, M.S., Head of Intelligence Alliances and Unification, Pharma Intelligence | Informa and Nancy Dvorin, Executive Editor, In Vivo

The past year saw intensifying dialogue on how to assess the value of and properly price therapeutics poised to cure diseases with a single dose, thus making drug pricing the major healthcare story of 2016. Stakeholders in the regenerative medicine space are preemptively laying the groundwork for the day when more of these products are approved and discussion of their cost-benefit analysis surfaces. With over 800 clinical trials involving regenerative medicine ongoing, and more than 60 trials in Phase III, this time will be approaching quickly.

As 2016 drew to a close, the U.S. Congress enacted the 21st Century Cures Act, which contains several provisions aimed at expediting the approval of advanced therapies products, including a designated pathway for regenerative advanced therapies (RAT). Drugs may qualify for the RAT designation if they are intended to treat or cure serious, life-threatening conditions, and if preliminary clinical evidence indicates the drug has the potential to address unmet medical needs. Further, FDA reorganized in October 2016, creating the Center for Biologics Evaluation and Research Office of Tissues and Advanced Therapies (CBER-OTAT), to consolidate and streamline its departments in an effort to make reviews of advanced regenerative medicine candidates more efficient.

On the European regulatory front, the European Medicines Agency issued an action plan that provides regulatory and scientific support for advanced therapy medicinal products, and prioritizes solutions that can be implemented quickly.

With these recent regulatory advances, combined with a new U.S. president and a new FDA commissioner, 2017 may see further acceleration in the approval process, especially for drugs intended to treat rare diseases.

In the clinical trials arena, 2016 was an exciting time for regenerative medicine companies seeking to advance their clinical programs. There were 21% more trials underway by year-end 2016 than the previous year, with 68% moving into either Phase II or III. Fate, Sangamo, Spark, Kiadis, Kite and many others had their therapies progress into Phases I and I/II for a variety of indications including cytomegalovirus, beta thalassemia, hemophilia B and a wide range of oncology indications. Phase II starts included Caladrius and Argos, to name a few. Companies with more advanced therapies—BioCardia, Adaptimmune, bluebird and others—initiated Phase III studies in 2016 for beta thalassemia, congestive heart failure, diabetic neuropathy and cancer indications.

Dealmaking in regenerative medicine was as strong as ever in 2016, with some notable strategic alliances, both cross-industry and industry-academia. Total global financing for this sector topped USD 5 billion, almost equally split between cell therapy and gene and gene-modified cell therapy areas. Big pharma companies Allergan and Pfizer acquired cell and gene therapy-focused biotechs in 2016. In a noticeable shift in financing from the previous year, corporate partnerships doled out less in upfront payments for their partnership deals (USD 850 million in 2016 vs. USD 2.3 billion in 2015) and added milestones as better indicators of value.

Nine regenerative medicine companies made initial public offerings during the year, raising more than USD 570 million between them with the majority focused on gene therapies. The companies included CRISPR Therapeutics, Editas Medicine, and Intellia Therapeutics, all focused on gene-editing. There were a number of innovative early-stage financings as well, with December’s USD 225 million deal between Bayer and Versant Ventures to form BlueRock Therapeutics, a next-gen stem cell play, being the biggest Series A closed in 2016. Advantagene, Topas Therapeutics and Eyevensis all completed Series A funding as well. Major partnerships in 2016 with substantial upfront payments included Biogen and UPenn, Pfizer and Bamboo Therapeutics, Adaptimmune and GSK, bluebird bio and Medigene, and Baxalta and Precision Biosciences.

2016 saw an ever-increasing pace of scientific advancement coupled with high expectations for novel therapies from the cell and gene therapy and tissue regeneration sectors. Investors and pharma alike continued to show interest in partnerships and investment in the sector. In the coming year, we hope to see a unified regulatory policy, potential solutions revolving around models of payers and reimbursement to ensure patient access to novel advanced therapies, and continuing financial support for this growing and dynamic advanced therapy arena. We look forward to continuing our analysis of regenerative medicine and advanced therapies throughout 2017.

To hear more on developments in the advanced therapies sector, and to meet and forge partnerships with decision makers from pharma, biotech and finance and patient advocacy groups, attend the Cell & Gene Exchange event taking place in Washington, DC, May 22–23, 2017.

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